Can a new drug ease nerve pain in rare heart disease?
NCT ID NCT05023889
First seen Jun 25, 2026 · Last updated Jun 27, 2026 · Updated 1 time
Summary
This study tested the drug patisiran in 10 adults with a rare condition called ATTR amyloidosis that causes nerve damage. Participants received an IV infusion every 21 days for 24 months. Researchers measured changes in nerve function, autonomic symptoms, and quality of life to see if the drug could help control the disease.
What this could mean
Our plain-language read of the trial. This is informational only — not medical advice or a prediction.
Active substance
patisiran (also known as ONPATTRO)
What this could lead to
If successful, this could point toward a treatment that slows or improves nerve damage and quality of life in people with ATTR amyloidosis.
What could go wrong
This is a very small, early-phase study with only 10 participants, so results may not apply broadly. The drug also requires lifelong dosing and premedication, and side effects are possible.
Disclaimer
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This is a summary of
the original study
.
Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.
This is a summary of the original study . Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.
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Conditions
The condition(s) this trial relates to.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
Contacts and locations
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Locations
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Austin Neuromuscler Center/National Neuromuscular Research Institute
Austin, Texas, 78759, United States